Vertex Announces U.S. FDA Approval for KALYDECO® (ivacaftor) to Treat Eligible Infants With CF Ages 1 Month and Older
-First and only CFTR modulator approved for this age group-
“Treating the underlying cause of cystic fibrosis as early as possible is important, and this approval, the first for a CFTR modulator in this age group, means families will now have a medicine for eligible infants,” said
“As a physician caring for infants and children with cystic fibrosis, I see the importance of initiating therapies early in life that may slow disease progression,” said
The approval was supported by a cohort in the Phase 3, 24-week, open-label study to evaluate the safety, pharmacokinetics and pharmacodynamics of ivacaftor in subjects with CF who are less than 24 months of age and have an ivacaftor-responsive CFTR mutation. This cohort demonstrated a safety profile similar to that observed in older children and adults.
KALYDECO® was first approved in 2012 in the
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 88,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the early 30s.
About KALYDECO® (ivacaftor)
In people with certain types of mutations in the CFTR gene, the CFTR protein at the cell surface does not function properly. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. KALYDECO® (ivacaftor) was the first medicine to treat the underlying cause of cystic fibrosis in people with specific mutations in the CFTR gene.
INDICATIONS AND USAGE
KALYDECO (ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 1 month and older who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR gene) that is responsive to KALYDECO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if KALYDECO is safe and effective in children under 1 month of age.
IMPORTANT SAFETY INFORMATION
Before taking KALYDECO, patients should tell their doctor if they: have liver or kidney problems; are pregnant or plan to become pregnant because it is not known if KALYDECO will harm an unborn baby; and are breastfeeding or planning to breastfeed because is not known if KALYDECO passes into breast milk.
Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works. Patients should ask their doctor or pharmacist for a list of these medicines if they are not sure. Patients should especially tell their doctor if they take the antibiotics rifampin or rifabutin; seizure medications such as phenobarbital, carbamazepine, or phenytoin; St. John’s wort; antifungal medicines such as ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.
KALYDECO can cause dizziness in some people who take it. If patients experience dizziness, they should not drive or operate machines until symptoms improve.
Patients should avoid food or drink containing grapefruit while taking KALYDECO.
KALYDECO can cause serious side effects including:
High liver enzymes in the blood, which have happened in patients receiving KALYDECO. The patient’s doctor will do blood tests to check their liver before starting KALYDECO, every 3 months during the first year of taking KALYDECO, and every year while taking KALYDECO. For patients who have had high liver enzymes in the past, the doctor may do blood tests to check the liver more often.
Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of their skin or the white part of their eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine.
Abnormality of the eye lens (cataract), which has happened in some children and adolescents receiving KALYDECO. The patient’s doctor should perform eye examinations before and during treatment with KALYDECO to look for cataracts.
The most common side effects include headache; upper respiratory tract infection (common cold), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.
These are not all the possible side effects of KALYDECO.
Use of KALYDECO in patients aged 1 month to less than 6 months born from a pregnancy lasting (gestational age) less than 37 weeks has not been evaluated.
Please click here to see the full Prescribing Information for KALYDECO.
Founded in 1989 in
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr.
International: +44 20 3204 5275